For the first time ever, there is substantial HOPE for children affected by Batten disease.

In my previous journal post ‘A Little Music’ I failed to include two important medical updates. One, we have joined forces (since Jan, 2012) with Noah’s Hope and Partnership for Cures in an effort to streamline our foundations’ grants to go toward research specific to Late Infantile Batten disease. When we attended the Batten

Disease Support and Research Association (BDSRA.org) conference this summer in Charlotte, we celebrated the fact that some of the research we supported (esp Noah’s Hope) was used in a study by Biomarin, a research phrameceutical company that develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. They developed a synthetic enzyme for treatment of LI Batten with regards to slowing or halting the progression of the disease. Their clinical trial is going to begin recruiting soon for the actual trial in 2013. This is so promising for future Batten families!!!! And two, there has been a press release about a study right here in Chicago at Rush University. They have discovered a way to actually produce the missing enzyme our kids lack with the use of Vitamin A and other lipid lowering FDA approved medications. This is absolutely incredible! Now the research needs to move toward clinic and ultimately trial for children suffereing from LI Batten. Please visit this link to read more about the breakthrough research in our field.http://www.24-7pressrelease.com/press-release/scientists-identify-drugs-for-batten-disease-304304.php